Lamzede is the first and only enzyme replacement therapy intended for the treatment of non-central nervous system manifestations of alpha-mannosidosis, an ultra-rare progressive disease with a wide range of symptoms
FDA approves Lamzede for rare genetic condition. (Credit: Arek Socha from Pixabay)
Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici, has received the US Food and Drug Administration (FDA) approval for Lamzede (velmanase alfa-tycv).
The drug is indicated for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and paediatric patients.
AM is an ultra-rare, progressive lysosomal storage disorder that occurs due to the deficiency of the α-mannosidase, an enzyme that works to split the alpha form of mannose.
In 2018, Chiesi received the European Commission (EC) marketing authorisation for Lamzede to treat non-neurological manifestations in patients with mild to moderate AM.
Chiesi Global Rare Diseases head Giacomo Chiesi said: “Lamzede is the first and only enzyme replacement therapy approved for alpha-mannosidosis in the United States, an achievement based on years of clinical development, as well as the dedication of our employees, clinicians, patients and their families.
“Alpha-mannosidosis presents with a variety of symptoms including impaired hearing, speech and mobility that progress from childhood into adulthood and is often under-recognised, causing some patients to be left undiagnosed or untreated.
“Lamzede is designed to provide an exogenous source of the α-mannosidase enzyme and we look forward to offering this medicine to patients in the United States who are eagerly awaiting a treatment option.”
According to Chiesi Global Rare Diseases, AM results in the improper breakdown of certain groups of complex sugars, leading to the accumulation of sugars in the body.
The build-up of sugars can affect several organs and organ systems of the body, and its effects may differ significantly from person to person, over time.
Chest and ear infections, hearing loss, distinctive facial features, muscle weakness, skeletal and joint abnormalities, visual abnormalities, and cognitive abnormalities, include symptoms.
Chiesi said that Lamzede is a recombinant form of human alpha-mannosidase and provided or supplements natural alpha-mannosidase, to prevent the accumulation of sugars.
Furthermore, the drug comes with a label warning about severe hypersensitivity reactions including cyanosis, emesis, urticaria, erythema, facial swelling, pyrexia, and tremor.